HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

Regimen F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: ABBV-CLS-7262 Dose 1; Drug: ABBV-CLS-7262 Dose 2; Drug: Matching Placebo

Detailed Description

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.

If a participant is randomized to Regimen F ABBV-CLS-7262, the participant will complete a screening visit to assess additional Regimen F eligibility criteria.

Once Regimen F eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in an overall 3:1 ratio to either active ABBV-CLS-7262 or matching placebo. The first 240 participants will be assigned in a 2:1:1 allocation ratio to Dose 1 ABBV-CLS-7262, Dose 2 ABBV-CLS-7262, or placebo. The final approximately 60 participants will be assigned in a 3:1 allocation ratio to Dose 1 ABBV-CLS-7262 or placebo.

Regimen F will enroll by invitation, as participants may not choose to enroll in Regimen F. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen F.

For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

• Study Type: Interventional
• Enrollment (Actual): 310
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Healey Center for ALS at Mass General

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 100 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria:

• The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

  1. Based on the metabolism of the compound, the concomitant use of certain inhibitors and inducers of cytochrome P450 enzymes.
  2. Any clinically significant ECG abnormalities.
  3. Clinically significant clinical laboratory abnormalities.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Matching Placebo	Drug: Matching Placebo; Matching placebo is administered orally once per day for 24 weeks.
Experimental: ABBV-CLS-7262 Dose 1	Drug: ABBV-CLS-7262 Dose 1; ABBV-CLS-7262 is administered orally once per day for 24 weeks.
Experimental: ABBV-CLS-7262 Dose 2	Drug: ABBV-CLS-7262 Dose 2; ABBV-CLS-7262 is administered orally once per day for 24 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease Progression as Assessed by the ALSFRS-R-Slope	Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Participants with higher scores have more physical function. Note that only participants who survived to their Week 24 visit contribute to the estimate.	Baseline to 24 Weeks
Mortality Event Rate	Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.	Baseline to 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Respiratory Function	Change in respiratory function over time as measured by Slow Vital Capacity (SVC).	Baseline to 24 Weeks
Function by ALSFRS-R Total Score	Change from baseline to Week 24 in function as assessed by ALSFRS-R total score.	Baseline to 24 weeks
Upper Limb Muscle Strength	Change in upper limb muscle strength over time as measured isometrically using hand-held dynamometry and grip strength, calculated as the average percent change from baseline of the following muscles/maneuvers: shoulder flexion, elbow flexion, elbow extension, wrist extension, abductor pollicis brevis contraction, abductor digiti minimi contraction, first dorsal interosseous contraction, and grip strength. Note that only those with measurable strength at baseline were included.	Baseline to 24 weeks
Disease Progression Biomarker	Change in log-transformed serum neurofilament light protein (NfL) concentration from baseline to Week 24.	Baseline to 24 Weeks
Activities of Daily Living	Change from baseline to Week 24 in the activities of daily living (ADL)/independence domain score as assessed by the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40). The ALSAQ-40, a patient-self reported outcome, consists of 40 questions that are used to measure the subjective well-being of participants, and each question is scored from 0 (never) to 5 (always or cannot do at all). The ADL/independence domain score is based on 10 out of the 40 questions, with a maximum ADL/independence domain score of 50 and minimum score of 0. Higher domain scores indicate a worse subjective well-being or less independence completing ADLs.	Baseline to 24 weeks
Number of Participants That Experienced Death or Death Equivalent	The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.	Baseline to 24 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Respiratory Function	Change in respiratory function over time as measured by Slow Vital Capacity (SVC).	24 Weeks

Collaborators and Investigators

• Sponsor: Merit E. Cudkowicz, MD
• Collaborators: Calico Life Sciences LLC
• Investigators: Principal Investigator: Merit Cudkowicz, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 23, 2023
• Primary Completion (Actual): October 3, 2024
• Study Completion (Actual): October 3, 2024

Study Registration Dates

• First Submitted: February 12, 2023
• First Submitted That Met QC Criteria: February 12, 2023
• First Posted (Actual): February 23, 2023

Study Record Updates

• Last Update Posted (Estimated): November 14, 2025
• Last Update Submitted That Met QC Criteria: October 31, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: ALS; Double-Blind; Placebo-Controlled; Lou Gehrig's Disease; Regimen Specific Appendix; ABBV-CLS-7262; Calico Life Sciences
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 2019P003518F

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No